PI3K Inhibitors For Hematology Indications Need Randomized Data, FDA Panel Says

21 Apr 2022

Vote can be seen as an endorsement of the Oncology Center of Excellence’s push for use of a single, ongoing randomized trial to serve as the basis for both accelerated approval and confirmatory evidence. OCE director Richard Pazdur says agency will be flexible in applying randomization requirements to future drugs that have a cleaner safety profile.

Crystal ball — ODAC members are not fortune-tellers, but FDA asked them to set development expectations for future PI3K inhibitors based on past experience with the class. • Source: Alamy

The US Food and Drug Administration’s external experts on 21 April backed the agency’s view that future approvals of phosphatidylinositol 3-kinase (PI3K) inhibitors for hematological malignancies should be based on randomized, rather than single-arm, studies.

The vote followed the assurance of Oncology Center of Excellence director Richard Pazdur that the agency would exercise flexibility around...